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Acasti Pharma Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was incorporated in 2002 and is headquartered in Laval, Canada.

Alpine Immune Sciences, Inc. operates as a clinical-stage biopharmaceutical company. It focuses on the discovery and development of immunotherapies through protein engineering technologies for treating autoimmune and inflammatory diseases. The company's product pipeline includes Povetacicept, a dual B cell cytokine antagonist, which is in Phase 2 clinical trial for the treatment of multiple autoimmune and inflammatory diseases; and Acazicolcept, a dual inhibitor of the CD28 and ICOS T cell costimulatory pathways that is in preclinical trial for the treatment of systemic lupus erythematosus. It is also developing RUBY-3 that has completed Phase 1 clinical trial for the treatment of glomerulonephritis; and RUBY-4, which has completed Phase 1 clinical trial for the treatment of cytopenias. The company was incorporated in 2007 and is headquartered in Seattle, Washington.

Enlivex Therapeutics Ltd. operates as a clinical-stage macrophage reprogramming immunotherapy company. It is developing Allocetra, a cell-based therapy to treat organ dysfunction and failure associated with sepsis that is in phase II clinical trial, as well as in preclinical trial to treat solid tumors. The company has a collaboration agreement with BeiGene to evaluate the safety and efficacy of Allocetra in combination with tislelizumab, an anti-PD-1 immune checkpoint inhibitor for the treatment of patients with advanced-stage solid tumors. Enlivex Therapeutics Ltd. is headquartered in Ness Ziona, Israel.

Stealth BioTherapeutics Corp, a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of therapies for diseases involving mitochondrial dysfunction. Its lead product candidate is Elamipretide that is in phase 3 clinical trial to treat barth syndrome, phase 2/3 clinical trial for the treatment of duchenne cardiomyopathy, phase 2a clinical trial to treat friedreich's ataxia, phase 2 clinical trial for the treatment of dry age-related macular degeneration, phase 2 clinical trial to treat leber's hereditary optic neuropathy, and phase 3 clinical trial for the treatment of replisome myopathies. The company's product candidates also include SBT-20 to treat complications of diabetes, renal diseases, ocular diseases, neurodegenerative, Parkinson's, Alzheimer's, Huntington's, and ALS diseases; SBT-550 for rare neurological indications; and SBT-272, a peptidomimetic that targets the mitochondria and stabilizing mitochondrial function under conditions of oxidative stress, as well as SBT 550. Stealth BioTherapeutics Corp was incorporated in 2006 and is based in George Town, the Cayman Islands.

Synlogic, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of synthetic biotics to treat metabolic diseases in the United States. Its pipeline include SYNB1618, an orally administered, non-systemically absorbed drug candidate to treat phenylketonuria; SYNB1934, an orally administered, non-systemically absorbed drug candidate, which is in Phase III clinical trial to treat phenylketonuria; SYNB1353, an orally administered, non-systemically absorbed drug candidate, which is in Phase I clinical to treat homocystinuria; SYNB8802, an orally administered, non-systemically absorbed drug candidate that is in Phase II clinical trial for the treatment of enteric hyperoxaluria; and SYNB2081 to lower uric acid for the potential treatment of gout. The company has a collaboration agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. for the research and pre-clinical development of a synthetic biotic medicine for the treatment of inflammatory bowel disease; and Ginkgo Bioworks, Inc. Synlogic, Inc. is based in Cambridge, Massachusetts.